Gender
All
Age Group
4 Years to 40 Years
Accepting Healthy Volunteers
No
Inclusion Criteria:
1. Males and females 4- ≥40 years of age with a clinical diagnosis of T1D
2. T1D clinical diagnosis with insulin start date no more than 100 days prior to the
time of randomization
3. Random non-fasting C-peptide level of >0.2 pmol/mL (equivalent to >0.6ng/ml) at
screening.
4. Positive for any one of the following diabetes-related autoantibodies (IAA, GAA,
IA-2, or ZnT8)
5. Treatment naïve of any immunomodulatory agent
6. Normal hearing at screening, defined as acceptable results of pure-tone audiometry
(<20 decibel [dB] baseline thresholds for frequencies 250, 500, 1000, and 2000 Hz
Exclusion Criteria:
1. Presence of severe, active disease that interferes with dietary intake or requires
the use of chronic medication, with the exception of well-controlled hypothyroidism
and mild asthma not requiring oral steroids. Presence of any psychiatric disorder
that will affect ability to participate in study.
2. Diabetes other than T1D
3. Chronic illness known to affect glucose metabolism (e.g. Cushing syndrome,
polycystic ovarian disorder, cystic fibrosis) or taking medications that affect
glucose metabolism (e.g. steroids, metformin)
4. Inability to swallow pills
5. Psychiatric impairment or current use of anti-psychotic medication
6. Any condition that, in the investigator's opinion, may compromise study
participation or may confound the interpretation of the study results.
7. Neutropenia (< 1,500 neutrophils/μL)
8. Leukopenia (< 3,000 leukocytes /μL)
9. Lymphopenia ( < 800 lymphocytes/μL)
10. Thrombocytopenia (<100,000 platelets/μL)
11. Clinically significant anemia or Hemoglobin as defined below:
In Adults: Hgb <12.0g/dL in females and <13.0g/dL in males In Children: 12- <18:
<11.4 g/dL in females and <12.4 g/dL in males In Children: 4- <12: Hgb <11.2 g/dL
12. Impaired renal function (assessed by history and BUN/Creatinine, DFMO is renally
excreted)
13. Allergy to milk or soy (components of Boost® drink used for mixed meal tolerance
testing)
14. Female participants of child-bearing age with reproductive potential, must not be
pregnant and agree to use 2 effective forms of birth control or be abstinent during
the study period (see below). Male participants (including men who have had
vasectomies) whose partners are pregnant or may be pregnant should use condoms while
on study drug, until 2 weeks after discontinuation of drug, while the partner is
pregnant.
15. Active seizure disorder, defined as requiring chronic medication at the time of
study or having had a seizure within the past 12 months at the time of screening
16. Enrollment into another intervention trial.
